Acadia, Stoke Team Up in $60M Deal to Develop RNA-based Therapies

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by Yedida Y Bogachkov PhD |

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Acadia Pharmaceuticals and Stoke Therapeutics are teaming up to develop and commercialize new RNA-based therapies — using Stoke’s proprietary research platform, TANGO — for the potential treatment of Rett syndrome.

As part of the collaboration, Stoke will receive a $60 million upfront payment from Acadia.

“Stoke’s RNA-based approach to upregulating healthy proteins offers very exciting new possibilities for the treatment of rare, neurodevelopmental diseases like Rett syndrome,” Steve Davis, CEO of Acadia, said in a press release.

“Combining Stoke’s capabilities with Acadia’s extensive expertise in neuroscience drug development and commercialization enables us to push harder and faster in exploring some of the new frontiers in rare central nervous system disorders,” Davis said.

Rett syndrome is mainly caused by a genetic mutation in the MECP2 gene, which is located on the X chromosome and codes for the MeCP2 protein. That protein regulates the activity of other genes by switching them off or on, playing an important role in brain development and function.

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The rare disorder almost exclusively affects girls. It impacts brain function, resulting in cognitive, emotional, sensory, and motor disturbances, as well as heart, breathing, and digestive issues.

Under the new deal, Stoke will lead research and preclinical development with the use of its proprietary research platform, TANGO — for Targeted Augmentation of Nuclear Gene Output. Stoke intends to use its technology to develop new treatments for diseases in which one copy of a gene functions normally, and the other is mutated, as is the case in Rett syndrome.

Its researchers will use TANGO and an understanding of RNA to create antisense oligonucleotides (ASOs), the company said.

ASOs are small pieces of DNA that can bind to specific molecules of messenger RNA — which have a well-known role in carrying instructions from DNA in protein production.

They are designed to bind and affect pre-mRNA, which is messenger RNA that still contains both coding and non-coding regions prior to modification. The idea is that ASOs can potentially help the single functional gene copy produce more protein, compensating for the mutated copy.

Acadia will be fully funding the research and preclinical development. It also will lead clinical development and commercialization activities with exclusive worldwide licenses for the Rett program.

In addition to Rett, the collaboration between Acadia and Stoke includes SYNGAP1 syndrome — a rare neurological disorder characterized by intellectual disability — and an undisclosed neurodevelopmental indication.

“We are excited to have the opportunity to further build our Rett syndrome franchise and pursue treatments in SYNGAP1 syndrome and other neurodevelopmental disorders,” Davis said.

Under the terms of the collaboration, Stoke now receives the $60 million, and has the potential to earn milestone payments of up to $907 million, as well as royalties on future sales of potential future treatments.

“Acadia shares our deep commitment to improving outcomes for people living with neurodevelopmental disorders,” said Edward M. Kaye, MD, Stoke’s CEO.

“We believe their late-stage development and commercialization capabilities, in addition to their neuroscience expertise, complement our discovery research efforts and clinical learnings,” Kaye said, adding, “Together, we believe we have a significant opportunity to improve treatment options by delivering new disease-modifying medicines to people who need them.”